A phase II, prospective, open label study (PO-MMM-PI-0011) to determine the safety and efficacy of pomalidomide (CC-4047) in subjects with primary, post polycythemia vera, or post essential thrombocythemia myelofibrosis (PMF post-PV MF, or post-ET MF)
The goal of this clinical research study is to learn if CC-4047 (now called pomalidomide) and prednisone can help to control MMM. The safety of this therapy will also be studied.
Disease Group: Leukemia,Myeloproliferative Diseases
Treatment Agent: CC 4047,Prednisone
Treatment Location: Only at MD Anderson
Estimatated Length of Stay in Houston: N/A
Return Visit: Study visits will occur at the end of Cycles 1,2,3 and 6, then can be extended to every 6 cycles if patient hasn't had any Grade 3-4 toxicity during the previous 3 cycles.
Home Care: N/A
Primary: · To determine the effect of CC-4047 and prednisone in the treatment of Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF, post-PV MF, or post-ET MF) · To determine the safety of CC-4047 and prednisone in the treatment of Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF, post-PV MF, or post-ET MF) Secondary: To further explore the nature and quality of responses to CC-4047 and prednisone
IRB Review and Approval Date: 12/04/2007
Recruitment Status: Closed
Projected Accrual: N/A
1) Must be >/= 18 years of age at the time of voluntarily signing an
Institutional Review Board/Independent Ethics Committee (IRB/IEC) –
approved informed consent form.
2) Must be diagnosed with myelofibrosis requiring therapy including myelofibrosis with myeloid metaplasia (MMM), de novo presentation (i.e. agnogenic myeloid metaplasia [AMMM], and developing after an antecedent history of Polycythemia vera (i.e., post-polycythemic myeloid metaplasia [PPMM]), or essential Polycythemia (i.e., post thrombocythemic myeloid metaplasia [PTMM]).
3) Screening total hemoglobin level < 10 g/dL or transfusion-dependent anemia defined as per IWG criteria (transfusion dependency defined by a history of a least 2 units of red blood cell transfusions in the last 28 days for hemoglobin < 8.5 g/dL that was not associated with overt bleeding)
4) Must have adequate organ function as demonstrated by the following </= 14 days prior to starting study drug: ·ALT (SGOT) and AST (SGPT) </= 3 x upper limit of normal (ULN), [unless upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis (EMH)] ·Total bilirubin < 3 x ULN or Direct Bilirubin < 2 x ULN ·Serum creatinine </= 2.5 mg/dL ·Absolute neutrophil count >/= 1,000/µL (>/=1.0 x 10^9/L) ·Platelet count >/= 50,000/µL (>/=50 x 10^9/L)
5) Subjects must be willing to receive transfusion of blood products
6) ECOG performance status (PS) of 0, 1, or 2 at screening.
7) Must be willing to adhere to the study visit schedule and other protocol requirements.
8) No active malignancies with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma (in situ) of the cervix or breast
9) All study participants must be registered into the mandatory POMALYST REMS™ program, and be willing and able to comply with the requirements of the POMALYST REMS™ program.
10) Females of reproductive potential (FCBP†) must adhere to the scheduled pregnancy testing as required in the POMALYST REMS™ program. Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use warfarin or low molecular weight heparin).
1) Known positive status for HIV, hepatitis B carrier, or active
hepatitis C infection.
2) The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea), corticosteroids, or experimental drug or therapy within 14 days of starting CC-4047 and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.
3) Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
4) Pregnant or lactating females
5) Prior use of CC-4047
6) Currently enrolled on another clinical trial or receiving investigational agent
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